Another week, another RNAi delivery breakthrough for the Alnylam/MIT partnership. Last week, the Cambridge, MA-based company and its academic partner published new data describing a new approach for systemic delivery of RNAi therapeutics using "lipidoids" This week, they're approaching delivery of gene-silencing RNA using what they're calling "core-shell" nanoparticles.
"Continued progress in delivery of RNAi therapeutics requires broad-based efforts around novel lipids, conjugates and polymers. In the current study, core-shell nanoparticles were discovered using combinatorial approaches to identify novel materials for siRNA delivery," Alnylam's Kevin Fitzgerald said in a statement. "These findings further expand our systemic delivery platform to achieve the broadest applications of RNAi therapeutics."
What they did to arrive at the core shells was evaluate more than 1,500 nanoparticles with diverse chemical properties, size and architecture. All these elements make a big difference in whether a nanoparticle works for drug delivery and whether there might be issues with toxicity. They found one type of nanoparticle that showed silencing of hepatocyte-specific Factor VII in an in vivo pre-clinical model.
Gene-silencing siRNA technology has failed to live up to its original hype largely because of problems delivering short interfering RNA to the precise spot where it's needed. Alnylam and MIT are experimenting with multiple ways of dealing the the problem.